Assessment of Macrophage Activation syndromE in STill's Disease in Italy

NCT06992505 | Completed

• 1 other identifier: Sobi.EMAPALUMAB-106
• Study Type: observational
• Target: 32
• Locations: 1 country
• Sites: 2

Brief Summary

Assessment of macrophage activation syndrome in Still's disease: retrospective chart analysis of patient history, symptom resolution and treatment characteristics in Italy

Conditions

Macrophage Activation Syndrome (MAS); Still Disease, Juvenile Onset; Still's Disease, Adult-Onset

Outcome Measures

Primary Outcomes (9)

• Overall survival

  defined as the survival time of patients from the index date

  from 2012 to 2022 (data abdstraction period)

• Time to laboratory value normalization

  defined as the time to normalization for key laboratory values

  up to 24 weeks

• Time to MAS laboratory remission

  defined as the time from index date to the last date of normalization of key laboratory assessments

  up to 24 weeks

• Time to partial MAS laboratory remission

  defined as the time from index date to the last date of normalization of at least 3 of the key laboratory assessments

  up to 24 weeks

• Time to tapering of GCs

  defined as the time from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose

  from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose

• Number of recurrent MAS episodes

  defined as the number of MAS episodes occurring any time after the end of the data collection period for the index MAS episode (hospital discharge or 26-weeks from the index date, whichever occurs later) until the end of study data collection

  from hospital discharge or 26-weeks from the index date, whichever occurs later, until the end of study data collection]

• Administration of organ support care

  defined as the proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs at any time from the index date until either the hospital discharge or 26 weeks, whichever occurs later

  from the index date until either the hospital discharge or 26 weeks, whichever occurs later

• Characteristics of MAS treatment

  Concomitant medications

  up to 24 weeks

• Clinical signs

  Patients experiencing MAS symptoms if present (documentation in free text)

  at index date and 8 weeks after index date (allowed time window 6 to 12 weeks)

Secondary Outcomes (6)

• Time to hospital discharge

  up to 24 weeks

• Time to intensive care unit (ICU) discharge

  up to 24 weeks

• Normalization of key laboratory values

  Week 8 after index date (allowed time window 6 to 12 weeks)

• MAS laboratory remission

  at Week 8 after index date (allowed time window 6 to 12 weeks).

• Partial MAS laboratory remission

  at Week 8 after index date (allowed time window 6 to 12 weeks)

Eligibility Criteria

• Age: 6 Months - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.

You may qualify if:

• Age \>6 months and ≤80 years at the beginning of the index MAS episode.
• Diagnosis of Still's disease (sJIA or AOSD diagnosis).
• Diagnosis of MAS according to treating physician in the medical record.
• Patients who have received at least 3 consecutive days of GC after diagnosis of MAS and/or are judged by the Investigator to be refractory to GC due to clinical worsening of patient's condition.
• The onset of the index MAS episode occurred between 01 January 2012 and 30 September 2022.
• According to local regulations, waivers of consent will be sought for study participants from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). o For patients not covered by waivers of consent, signed, and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable) or who have died, should be obtained before any study-related activities are undertaken.

You may not qualify if:

• A diagnosis of primary HLH prior to the beginning of the index MAS episode.
• Confirmed malignancy prior to the beginning of the index MAS episode.
• Patient treated with any investigational product as a part of clinical trial during the index MAS episode.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (2)

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Ospedale Pediatrico Bambin Gesù

Rome, 00165, Italy

Location

MeSH Terms

Conditions

Macrophage Activation Syndrome; Arthritis, Juvenile; Still's Disease, Adult-Onset

Condition Hierarchy (Ancestors)

Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Arthritis, Rheumatoid

Study Officials

• Study Physician

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2025
• First Posted: May 28, 2025
• Study Start: June 6, 2025
• Primary Completion: November 27, 2025
• Study Completion: November 27, 2025
• Last Updated: December 2, 2025

Record last verified: 2025-11

Locations

IT (2)